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Oral Pyrophosphate Absorption in PXE Disease

Primary Purpose

Pseudoxanthoma Elasticum

Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Disodium Pyrophosphate
Sponsored by
Tampere University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pseudoxanthoma Elasticum

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Adults (>18 yrs)
  • clinically and genetically proven PXE
  • Body mass index (BMI) 18,5-34,9 kg/m2

Exclusion Criteria:

  • Special groups according to researchers' decision.
  • Pregnancy
  • No effective contraception in females in child-bearing age.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Open label

    Arm Description

    Disodiumpyrophosphate, capsuled powder, First day: 30 mg/kg fasting at 08.00 and with standard mixed meal at 12.00 Second day: 50 mg/kg fasting at 08.00 and with standard mixed meal at 12.00

    Outcomes

    Primary Outcome Measures

    Concentration area under the curve 0-t of pyrophosphate
    Maximal concentration of pyrophosphate
    Cmax
    Timepoint of maximal pyrophosphate concentration
    Tmax

    Secondary Outcome Measures

    Concentration area under the curve 0-t of phosphate
    AUC0-t
    Maximal concentration of phosphate
    Cmax
    Timepoint of maximal phosphate concentration
    Tmax

    Full Information

    First Posted
    January 20, 2020
    Last Updated
    April 21, 2021
    Sponsor
    Tampere University Hospital
    Collaborators
    UMC Utrecht, Hungarian Academy of Sciences
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04441671
    Brief Title
    Oral Pyrophosphate Absorption in PXE Disease
    Official Title
    Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE)
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2021
    Overall Recruitment Status
    Withdrawn
    Why Stopped
    Withdrawn
    Study Start Date
    December 8, 2020 (Actual)
    Primary Completion Date
    April 1, 2021 (Actual)
    Study Completion Date
    April 1, 2021 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Tampere University Hospital
    Collaborators
    UMC Utrecht, Hungarian Academy of Sciences

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    Pyrophosphate is an endogenous, non-toxic metabolite inhibiting soft tissue calcification. The aim of our study is to find optimal dosing and safety of oral disodium-PPi (Na2H2PPi). Absorption curves (pharmacokinetics), AUC0-t, Cmax and Tmax for PPi and phosphate will be provided for healthy controls and PXE-patients both fasting and with standard meal intake.
    Detailed Description
    Phase II oral capsulized disodium-PPi (Na2H2PPi) powder absorption study in subjects with PXE (n=8-12) will be done in the ward of Internal Medicine of Tampere University Hospital and University Medical Center Utrecht. After a 10-hour fast at 8.00 a.m. 30 mg/kg (first day) and 50 mg/kg (second day) capsulized dose of PPi will be given with 2 dl water. At 12.00 a.m. another capsulized 30 mg/kg (first day) or 50 mg/kg (second day) single dose of PPi with a standard mixed meal (lunch) will be given with 2 dl water at the time when a subject starts eating. Plasma and urine sampling include plasma and spot urine electrolytes, creatinine, and pyrophosphate. Plasma sampling will be done at 0, 15, 30, 60, 120 and 240 min after ingestion of PPi. Urine spot sample will be taken at 0 and 240 min after ingestion of PPi. Physical activity is restricted. Side-effects will be recorded.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Pseudoxanthoma Elasticum

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Open label
    Arm Type
    Experimental
    Arm Description
    Disodiumpyrophosphate, capsuled powder, First day: 30 mg/kg fasting at 08.00 and with standard mixed meal at 12.00 Second day: 50 mg/kg fasting at 08.00 and with standard mixed meal at 12.00
    Intervention Type
    Drug
    Intervention Name(s)
    Disodium Pyrophosphate
    Other Intervention Name(s)
    Na2H2PPi
    Intervention Description
    Absorption trial
    Primary Outcome Measure Information:
    Title
    Concentration area under the curve 0-t of pyrophosphate
    Time Frame
    two days
    Title
    Maximal concentration of pyrophosphate
    Description
    Cmax
    Time Frame
    two days
    Title
    Timepoint of maximal pyrophosphate concentration
    Description
    Tmax
    Time Frame
    two days
    Secondary Outcome Measure Information:
    Title
    Concentration area under the curve 0-t of phosphate
    Description
    AUC0-t
    Time Frame
    two days
    Title
    Maximal concentration of phosphate
    Description
    Cmax
    Time Frame
    two days
    Title
    Timepoint of maximal phosphate concentration
    Description
    Tmax
    Time Frame
    two days

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    80 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Adults (>18 yrs) clinically and genetically proven PXE Body mass index (BMI) 18,5-34,9 kg/m2 Exclusion Criteria: Special groups according to researchers' decision. Pregnancy No effective contraception in females in child-bearing age.
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Pasi I Nevalainen, MD, PhD
    Organizational Affiliation
    Tampere University Hospital
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    No
    IPD Sharing Plan Description
    Only anonymized data can be shared

    Learn more about this trial

    Oral Pyrophosphate Absorption in PXE Disease

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